Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one ... Read more
Updates
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Amryt Releases Design of Pivotal Trial Evaluating Oleogel-S10 for EB Patients of All Ages
Amryt Pharma is conducting a Phase 3 clinical trial evaluating the effectiveness and long-term safety of Oleogel-S10 (AP101), a topical formulation of birch bark ... Read more -
Aegle Wins Debra’s Partners in Progress Award, Plans to Soon Open Clinical Trial in DEB Patients
With its lead therapy candidate AGLE-103 set to begin clinical testing in the coming months, Aegle Therapeutics is being honored with a Partners in Progress Award ... Read more -
Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of ... Read more -
Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in ... Read more
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Gene-modified Skin Cells Show Promise for RDEB in Phase 1 Trial
An experimental cell therapy where a patient’s own skin cells (fibroblasts) are engineered in ... -
Transplants Using Patients’ Own Skin May Be Safe, Effective Treatment for RDEB, Trial Suggests
Transplanting a patient’s own healthy skin to treat their skin ulcers was found to ... -
European Initiative Targets Diagnosis, Treatment of Rare Diseases
A new international consortium based in Paris, and funded largely by the 28-member European ... -
FDA Releases Guidelines to Push Industry Development of New Therapies
The U.S. Food and Drug Administration (FDA) has released a guidance document to assist ... -
Macedonian Gaucher Activist Publicizes Plight of Rare Disease Patients
The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow ... -
World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in ...
Factors Beyond COL7A1 Mutations May Contribute to Dystrophic EB Forms, Study Suggests
News
A study from Spain has raised the possibility that the development of dystrophic epidermolysis bullosa (DEB) could be attributable to additional factors besides mutations in the COL7A1 gene. Researchers reached this conclusion after studying three unrelated patients with different forms of ... Read more